A study looking at treatment for acute myeloid leukaemia and high risk myelodysplastic syndrome (AML18 Pilot)

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Cancer type:

Acute leukaemia
Acute myeloid leukaemia (AML)
Blood cancers
Myelodysplastic syndrome (MDS)




Phase 1/2

This pilot study was done to see if it’s possible to do a large trial looking at quizartinib, plerixafor and ganetespib with chemotherapy for people over 60.

More about this trial

This trial was for people who have acute myeloid leukaemia (AML) or high risk myelodysplastic syndrome (MDS). High risk MDS means that there is a high risk that it will develop into leukaemia.

Doctors usually treat AML and MDS with chemotherapy. Patients who are fit enough have intensive treatment over several months. The aim is to get rid of all the leukaemia cells, and stop them coming back. This trial was for people who were due to have intensive treatment.

When this trial was done, doctors often used one of these combinations of chemotherapy drugs to treat AML and MDS:

  • daunorubicin, Ara C and etoposide (DAE)
  • daunorubicin and clofarabine (DClo)

Researchers want to find out more about using 3 other drugs alongside chemotherapy. They are:

  • plerixafor, which may make leukaemia cells more sensitive to chemotherapy
  • quizartinib (AC-220), a type of cancer growth blocker called a tyrosine kinase inhibitor (TKI)
  • ganetespib, a type of cancer growth blocker called a heat shock protein 90 (HSP90) inhibitor

The aims of this pilot study were to:

  • see if it would be possible to look at these new treatments in a large trial
  • find the highest dose of quizartinib you can have alongside chemotherapy
  • see if any of these treatments help people with AML or MDS
  • learn more about the side effects

Summary of results

Everyone taking part in this trial had been diagnosed with either acute myeloid leukaemia (AML) or high risk myelodysplastic syndrome (MDS). They all had either DAE or DClo chemotherapy, alongside one of the drugs below.

The research team recruited 113 people between 2011 and 2013, and analysed the results in 2014.

There were 44 people in the plerixafor group. The first few had a low dose. And as they didn’t have any serious side effects, the next few had a higher dose. And so on. But the company that made the drug decided they didn’t want to continue with the trial, so the researchers stopped this part of the trial earlier than planned. And the main AML18 trial won’t include plerixafor.

There were 55 people in the quizartinib group. People in different groups had different treatment doses over either 1 week or 2 weeks. The trial team looked at the side effects people had to decide the best dose to give. Side effects included changes in heart rhythm and infection.

When they looked at how well the treatment worked they found that the leukaemia went away in more than 6 out of 10 people (65%).

The research team decided that quizartinib was safe to use and could be a useful treatment. They concluded that it should be part of the main AML18 trial.

There were 14 people in the ganetespib group. They all had the same dose of ganetespib.

The research team found that the leukaemia had gone away in 9 people (64%). And that nearly 8 out 10 people (79%) were living 6 months after treatment. This is slightly higher than the results of AML16, a large trial looking at treatment for AML. Side effects included diarrhoea and fever or infection.

The research team decided that ganetespib didn’t cause too many side effects and could be a useful treatment. They concluded that it should be part of the main AML18 trial.

The research team concluded that quizartinib and ganetespib should both be looked at in a larger trial, AML18. But the results from other trials for ganetespib were not as good, and the company who made it decided to stop developing it. So it is no longer part of the AML18 trial.

We have more information about the AML18 trial on our website.

We have based this summary on information from the research team.  As far as we are aware, the information they sent us has not been reviewed independently (peer reviewed) or published in a medical journal yet. The figures we quote above were provided by the research team. We have not analysed the data ourselves.

Recruitment start:

Recruitment end:

How to join a clinical trial

Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Chief Investigator

Professor Alan Burnett

Supported by

Cardiff University
Experimental Cancer Medicine Centre (ECMC)
NIHR Clinical Research Network: Cancer

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Last review date

CRUK internal database number:


Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Cara took part in a clinical trial

A picture of Cara

"I am glad that taking part in a trial might help others on their own cancer journey.”

Last reviewed:

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